In "Drug Truths: Dispelling the Myths About Pharma R&D," author John L. LaMattina, former head of global R&D at Pfizer, uses numerous examples from Pfizer, and a few from other drug companies, to convince—or simply remind—the reader that big pharma plays a most essential role in maintaining our health. To him, it's as clear as day that without big drug companies, too many of us would be dying long before our time or living lives that are so full of pain, debilitating diseases, and mental traumas as to be not worth continuing.

Big drug companies tend to be extremely cautious when making public statements because their audiences include the financial community, the Food & Drug Administration, patients, and their competitors. Spanning a mere 152 pages, the book is refreshingly straightforward in its defense of big-pharma R&D. Unsurprisingly, LaMattina was known while still at Pfizer as an uncharacteristically outspoken manager.

His book offers a series of rebuttals to the most common criticisms aimed at big-pharma R&D, starting with the notion that cholesterol drugs are unnecessary and ending with the idea that the business model followed by big drug companies is fatally flawed. The book is a worthwhile read for those who have already heard from big pharma’s critics but haven’t yet seen a well-articulated response from an industry insider.

LaMattina provides the reader with numerous, often surprising insights about what it’s like to be a researcher in a big drug company. For instance, the financial and pharmaceutical trade press routinely depicts Pfizer as a company that is nearly desperate to come up with a new drug that will generate as much revenue as Lipitor, a cholesterol drug. Lipitor has annual sales in excess of $10 billion, but it comes off patent in 2011. It may be true that Pfizer needs something to replace Lipitor, but LaMattina leaves the impression that the company’s researchers are not really that concerned whether their new treatments turn out to be the runaway commercial successes that top management might expect. As LaMattina tells it, the catchphrase among Pfizer’s researchers is “the patient is waiting.”

Through numerous case studies, LaMattina demonstrates that “the patient is waiting” is an apt description for what motivates researchers at Pfizer and other big drug firms. For instance, Pfizer discovered that its nerve pain treatment Lyrica (generic name pregabalin) could be used to treat fibromyalgia, a painful and debilitating condition that afflicts many patients after a surgical procedure. Until 2007, when FDA approved Lyrica for treating fibromyalgia, the condition was incurable. LaMattina says that until Lyrica proved itself as a treatment, people suffering from fibromyalgia were simply being told that “their disease was in their head.”

“It is hard to believe there won't be a resurgence of new drug approvals across multiple therapeutic areas in the coming years."

LaMattina is peculiarly ardent in his defense of Lipitor and other cholesterol-lowering drugs that many consider to be unnecessary. “For the press to challenge a key part of current treatment paradigms for heart disease borders on irresponsibility,” he writes. Statins, he adds, are one of the main reasons, along with improved diagnosis and understanding of the risk factors, for the steady lowering of cardiovascular deaths that has been occurring over the past 25 years.

This defense of cholesterol-lowering statins, appearing early on in the book, is somewhat unconvincing, however, and possibly the weakest part of the book. LaMattina refers to numerous studies that prove the effectiveness of statins but makes light of possible alternatives to taking these drugs, such as sticking to a regular exercise program and living on a vegetable-rich diet.

"Everyone—physicians, health care providers, employers, and governments—should be pushing people to do this," he writes. "But the fact is that the effect of the obesity-diabetes-heart disease progression is going to last for decades," he adds. LaMattina then proceeds to argue in favor of administering statins to people who don't exhibit any signs of heart disease, primarily because atherosclerosis, better known as hardening of the arteries, takes decades to develop and also because the first symptom of heart disease is often a heart attack. He does not discuss the risks and benefits of such an approach, even though some patients develop amnesia or muscle weakness as a result of taking statins. Rather, he argues that there are enough data demonstrating the benefits of administering statins to healthy people and that a more rigorous study "would be prohibitive to execute due to cost and scale."

LaMattina is far more effective in his rebuttal of the notion that the drug industry is excessively focused on developing me-too drugs. He shows that it is not a waste of research resources for different companies to develop nearly identical drugs that target a particular disease in nearly identical ways.

To start with, he writes, the nearly identical research programs at various companies get under way several years before any of the companies are ready for the commercial launch of a drug, usually after the publication of breakthrough fundamental research. Any, if not most, of these programs could fail. Next, because patients respond in dissimilar and unexpected ways to particular medicines, physicians are better off having several slightly different drugs to choose from. Pfizer’s Lipitor offered vast improvements over other statins when it was launched, for example, something that has been proven by numerous studies, LaMattina explains.

As he methodically tackles the most common “myths” circulating about big pharma R&D, LaMattina provides a collection of counterintuitive facts. In the chapter “Big Pharma Has Failed and Should Learn More from Biotech Success,” he shows readers that R&D productivity at major drug companies is not any lower than at biotech firms. Another unexpected fact, found in the chapter called “Big Pharma’s Day Has Passed,” is that the R&D pipeline of major drug companies is actually quite strong, even if an ever smaller number of drugs have been approved for launch by FDA in recent years. LaMattina reports that between 2002 and 2007, the number of drugs in Phase II clinical trials has surged from five to 15 at Merck and from 21 to 47 at Pfizer. “It is hard to believe there won’t be a resurgence of new drug approvals across multiple therapeutic areas in the coming years,” LaMattina writes.

The book does a great job of humanizing the drug discovery efforts of major drug companies. LaMattina provides several war stories of teams of researchers who have dedicated themselves for years to bringing a particular drug to patients. These researchers at times succeeded in their efforts, but more often have not. LaMattina describes several instances of these heartbreaking failures.

For example, when Pfizer halted a 17-year effort to develop a cholesteryl ester transfer protein inhibitor to treat heart disease, LaMattina reports a feeling of bereavement by the head of clinical trials for the program, Chuck Shear. “Something was gone that would never be replaced, a hole in my heart that will remain forever,” Shear said, according to LaMattina. This human tale is something we don’t get from the sanitized press releases and earnings reports that big companies normally issue to communicate with the public.

One of the questions left unanswered by this short book is how big drug companies like Pfizer prioritize their research commitments given that it would be too onerous to fund all possible research programs. The book discusses Pfizer’s work in developing treatments for cardiovascular diseases, pain, HIV, obesity, and tropical diseases but does not really explain why the firm is involved in these disparate therapeutic areas. Instead, one gets the impression that the company will fund any program that might yield a useful drug.

Despite some weaknesses, which are probably the result of it being so concise, the book provides unique insights into how big pharma manages its R&D. It also serves as a reminder of the useful role the drug industry plays in keeping us healthy. “Be it life-saving medicines to treat cancer, new drugs to treat painful arthritis that ease the lives of the elderly, or new antibiotics to treat a child’s ear infection, this industry is a source of hope,” LaMattina writes. “Only pharmaceutical R&D discovers, develops, manufactures, tests and demonstrates the properties of compounds that prove or disprove medical hyphotheses. Nowhere else is this done.”